An agency of the European Union

PRIority MEdicines (PRIME)

Support to development of priority medicines for unmet medical needs. EuropaBIO information day, 15 October 2015 Jordi Llinares, Head of product development scientific support .

Patients

Areas of unmet need Focus on accelerating

regulatory approval of new medicines

Research & Development

Scientific and regulatory challenges

Importance of early dialogue with regulators and scientific advice

Difficulty in access to capital investment for academia & SMEs

EU Network Perspective

Optimising support to innovation

Complementary approach to national initiatives

Supporting global development

Drivers for change

Reinforcing PREDICTABILITY of the EU regulatory system.

Vision of the EU Medicines Regulatory Network

EU Medicines Agencies Network Strategy to 2020

Ensure timely access to new beneficial and safe medicines for patients

- Better understanding of existing tools (conditional MA, accelerated assessment…) and

prospective planning of their use

Support for patient focused innovation and contribute to a vibrant life science sector in

Europe

- Facilitate innovation to ensure patient access to new medicines

- Greater collaboration across network to support innovation

- Consider further regulatory incentives for innovation, particularly in certain areas of

public health need

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According to Recital 33 and Article 14(9) of

Regulation (EC) No 726/2004, the applicant may

request an accelerated assessment procedure in

order to meet, in particular the legitimate

expectations of patients and to take account of the

increasingly rapid progress of science and

therapies, for medicinal products of major interest

from the point of view of public health and in

particular from the viewpoint of therapeutic

innovation.

Regulation (EC) No 726/2004

PRIME - Legal base

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Goal & Scope

To foster the development of medicines with high public health potential.

Reinforce scientific and regulatory advice

Foster and facilitate early interaction

Raise awareness of requirements earlier in development

Optimise development for robust data generation

Focus efficient development

Promote robust data generation

Enable accelerated assessment

Promote generation of high quality data

Facilitated by knowledge gained throughout development

? !

Building on

existing

framework;

Eligibility

according to

existing

Accelerated

Assessment

criteria.

Reinforcing the concept of Accelerated Assessment

Current approach

Confirmation of evaluation of a

centralised MAA to an accelerated

timetable has only been possible just

prior to filing.

With PRIME

Identifying products fulfilling the criteria

for accelerated review earlier

Enhancing the regulatory support on offer

to these products through advice at key

milestones in development

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Eligibility to PRIME scheme

Entry to scheme at two different stages in development:

at the earlier stage of proof of principle (prior to phase

II/exploratory clinical studies) focusing on SMEs.

at proof of concept (prior to phase III/confirmatory clinical studies).

Must be based on adequate data to justify a potential major public

health interest.

For products under development which are yet to be placed on the EU market.

Applicants not eligible to PRIME can still request accelerated assessment. Guideline of the procedure for Accelerated Assessment pursuant to Article 14(9) of Regulation (EC)

No 726/2004.

Enriched support through PRIME scheme

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Tailored to the stage of development and provided up to submission of MAA;

CHMP Rapporteur appointment for products at proof of concept stage;

Kick-off meeting with the Rapporteur and experts from relevant committees to discuss

development options and regulatory strategy;

Iterative scientific advice at major development milestones;

Fee incentives for SMEs throughout the development.

Benefits of the PRIME scheme

Early confirmation of potential for accelerated assessment;

Written confirmation of PRIME eligibility;

Timely CHMP Rapporteur appointment;

Scientific advice at key development milestones/decision points;

Early, proactive, continuous and strengthened regulatory support;

Promote awareness and better use of existing development and

authorisation tools;

EMA dedicated entry point;

Complementarity and collaboration with National innovation

schemes;

Fee incentives for SMEs on Scientific Advice requests.

Early access tool, supporting patient access to innovative medicines.

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Nonclinical Phase I Exploratory Confirmatory Evaluation Post-

authorisation

Early identification of therapeutic

innovation in unmet medical needs.*

MAA review under accelerated assessment.

Iterative Scientific advice

Enhanced regulatory guidance

Incremental knowledge gain

Proactive dialogue

Promote use of existing tools

* Based on existing Accelerated assessment criteria

Supporting patient access to innovative medicines

Nonclinical Phase I Exploratory Confirmatory Evaluation Post-

authorisation

Supporting patient access to innovative medicines

National scientific advice Early identification

of therapeutic innovation in unmet

medical needs.*

MAA review under accelerated assessment.

Iterative Scientific advice

Enhanced regulatory guidance

Incremental knowledge gain

Proactive dialogue

Promote use of existing tools

Eligibility (CHMP)

Accelerated Assessment confirmation (CHMP)

Any

sponsor

SA 1 (SAWP) SA 2

(SAWP) Full MA

Exceptional

Conditional

SA n (SAWP)

Early CHMP Rapporteur appointment

Early CHMP Rapporteur appointment SMEs

Timely approval and patient access to important

new medicines;

Builds on accelerated assessment criteria;

Promote better use of existing tools;

Optimise current regulatory tools by increasing

efficiency of development and quality of data;

Opportunity for collaboration with partners across

the EU network.

Launch

by q1

2016.

Summary

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Thank you for your attention

Jordi.Llinares@ema.europa.eu

European Medicines Agency

30 Churchill Place • Canary Wharf • London E14 5EU • United Kingdom

Telephone +44 (0)20 3660 6000 Facsimile +44 (0)20 3660 5555

Send a question via our website www.ema.europa.eu/contact

Further information

Follow us on @EMA_News

Adaptive Pathways

Conditional MA

PRIME

Accelerated Assessment

Early access tools: Overview

Scientific concept of development and data generation.

Iterative development with use of real-life data.

Engagement with other healthcare-decision makers.

Unmet medical need, seriously debilitating or life-threatening disease, a rare disease or use in emergency situations.

Early approval of a medicine on the basis of less complete clinical data.

Major public health interest, unmet medical need.

Dedicated and reinforced support.

Enable accelerated assessment.

Better use of existing regulatory & procedural tools.

Major public health interest, unmet medical need.

Reduce assessment time to 150 days.

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Adaptive Pathways

Conditional MA

PRIME

Accelerated Assessment

Early access tools: Ongoing activities

Pilot ongoing.

ADAPT-SMART

Ongoing revision of the guideline.

Emphasis on importance of prospective planning early dialogue.

New scheme.

Relevant Q&A, scientific guidance and templates under development

for launch

Ongoing revision of the guideline.

More detailed guidance on justification.

Optimisation of the assessment timetable.

Emphasis on the importance of early dialogue.

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